HFSA 2024: Cardiac myosin inhibitor improves symptoms in obstructive HCM

Aficamten significantly improved symptoms, hemodynamic response, exercise capacity, and biomarker levels in patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in secondary efficacy analyses of the SEQUOIA-HCM trial. These results were shared in a virtual late-breaking trial session at the Heart Failure Society of American meeting.

Nearly 300 symptomatic oHCM patients were randomized to receive either aficamten (n = 142) or placebo (n = 140) daily for 24 weeks. Primary outcomes included complete hemodynamic response, symptom relief, enhanced exercise capacity, and reduction in N-terminal pro-B-type natriuretic peptide levels. Eligibility for septal reduction therapy was also assessed.

After 24 weeks, an improvement in ≥1 of these outcome measures was achieved in 97% of patients treated with aficamten (vs. 59% placebo), including 23% on aficamten who achieved all 4 outcomes compared with none in placebo:

• Improvement in limiting symptoms (71% vs. 42%)
• Increased likelihood of a complete hemodynamic response (68% vs. 7%)
• Enhanced exercise capacity (47% vs. 24%)
• Reduction of ≥50% in N-terminal pro-B-type natriuretic peptide levels (84% vs. 8%; P ≤0.002 for all above).

Additionally, 88% of aficamten-treated patients eligible for septal reduction therapy were no longer eligible, compared with 52% in the placebo group (P = 0.002).

Source:

Maron MS, et al; SEQUOIA-HCM Investigators. (2024, September 25). J Am Coll Cardiol. Impact of Aficamten on Disease and Symptom Burden in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM. https://pubmed.ncbi.nlm.nih.gov/39352339/